Longitudinal Studies for Vasculitis

系统性脉管炎是一组以严重的血管炎症为特征的疾病。 如果不治疗，会导致动脉狭窄或闭塞，以及器官损伤或死亡。具有积极的免疫抑制作用 治疗后，大多数患者存活，但随后的病程各不相同，复发较多 预计脉管炎或其治疗会导致常见和长期的发病率。长期纵向研究 在这个管理适当但不完善的时代，需要向临床医生提供信息和指导。也有 未满足的重大需求：一)疾病活动性、复发风险和长期损害的生物标志物；二) 遗传流行病学研究，提供对发病机制的洞察和治疗思路；iii)早期阶段 新疗法的临床试验；四)进行高质量临床试验和临床试验的新方法 对这些罕见疾病的研究；以及v)改进结果衡量标准，包括患者报告 结果。VCRC纵向研究通过多项研究解决了所有这些未得到满足的需求 与这些方案相关的研究。 脉管炎临床研究联盟，在美国国立卫生研究院罕见疾病中心的支持下于2004年成立 临床研究网络(RDCRN)，包括对六种血管炎[巨细胞]的纵向队列研究 大动脉炎(GCA)、大动脉炎(TAK)、结节性多动脉炎(PAN)、肉芽肿合并多血管炎 (Wegener‘s，GPA)、显微镜下多血管炎(MPA)和嗜酸性肉芽肿合并多血管炎(Churg- Strauss，EGPA)]在美国和加拿大的领先转介中心工作。已有5000多名患者接受了 已注册。VCRC纵向研究是本RDCRC目标的核心，并作为 探索新的潜在疾病生物标记物的基金会，第二阶段和111项临床试验； 制定改进的结果衡量标准，培训脉管炎研究研究员，并与 病人支持组织。 该项目的具体目标是：1)支持VCRC继续成功地进行 六种脉管炎的纵向研究，2)扩大纵向研究以包括孤立性 以及3)维护和扩展VCRC生物医学和临床数据存储库，以供 范围广泛的科学家，包括联盟调查人员核心小组内外的科学家。

The systemic vasculitides are a group of diseases characterized by severe inflammation of blood vessels leading to arterial stenosis or occlusion, and organ damage or death if untreated. With aggressive immunosuppressive treatment, most patients survive, but subsequent disease course is variable, relapse is common, and long-term morbidity from vasculitis or its treatment is expected. Long-term longitudinal studies are needed to inform and guide clinicians in this era of adequate but imperfect management. There are also significant unmet needs for i) biomarkers of disease activity, risk of relapse, and long-term damage; ii) genetic epidemiology studies to provide insight into pathogenesis and ideas for therapeutics; iii) early-phase clinical trials of novel therapeutics; iv) novel methods to conduct high-quality clinical trials and clinical research studies in these rare diseases; and v) for improved outcome measures, including patient-reported outcomes. The VCRC Longitudinal Studies address all these unmet needs through the multiple research studies associated with these protocols. The Vasculitis Clinical Research Consortium, initiated in 2004 under the auspices of the NIH Rare Diseases Clinical Research Network (RDCRN), encompasses longitudinal cohort studies of six vasculitides [giant cell arteritis (GCA), Takayasu's arteritis (TAK), polyarteritis nodosa (PAN), granulomatosis with polyangiitis (Wegener's, GPA), microscopic polyangiitis (MPA), and eosinophilic granulomatosis with polyangiitis (Churg- Strauss, EGPA)] at leading referral centers in the US and Canada. More than 5000 patients have been enrolled. The VCRC Longitudinal Studies are central to the goals of this RDCRC and serve as the foundation for the exploration of new potential biomarkers of disease, phase II and 111 clinical trials, development of improved outcome measures, training of fellows in vasculitis research, and collaboration with patient support organizations. The Specific Aims of this project are to 1) support the continued successful conduct of the VCRC Longitudinal Studies of six vasculitides, 2) expand the Longitudinal Studies to include patients with isolated aortitis, and 3) maintain and expand the VCRC Biospecimen and Clinical Data Repositories for use by a broad range of scientists, both within and beyond the core group of Consortium investigators.